− Framework Ties Payment for GIVLAARI to its
Delivery of Patient Outcomes in the Real-World Setting -
− Includes New Approach Designed for Ultra-Rare
Diseases that Gives Participating Payers Greater Financial
Certainty if Disease Prevalence is Higher Than Anticipated –
- Discussions Underway with Leading Insurers
and Agreement in Principle in Place with Harvard Pilgrim -
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, is announcing today a new and enhanced
framework for value-based agreements (VBAs) designed to help
patients with acute hepatic porphyria (AHP) – an ultra-rare orphan
disease – gain access to GIVLAARI™ (givosiran) injection for
subcutaneous use, a first-of-its-kind RNAi therapeutic for the
treatment of AHP. Approved today by the U.S. Food and Drug
Administration (FDA), GIVLAARI is indicated for the treatment of
adults with AHP.
Alnylam is in active discussions with leading payers about VBAs
for GIVLAARI and plans to incorporate this new ultra-rare disease
framework into these discussions and negotiations. Alnylam has
reached an agreement in principle with Harvard Pilgrim covering
GIVLAARI.
Under this innovative framework for VBAs, participating
government and commercial payers will pay the full value for
GIVLAARI only when it delivers patient outcomes in the real-world
setting similar to results demonstrated in clinical trials. An
additional and newly designed Prevalence-Based Adjustment (PBA)
feature will trigger rebates to participating payers if the number
of diagnosed patients they cover exceeds current epidemiologic
estimates for AHP. There are often uncertainties in diagnosis rates
and disease prevalence estimates in ultra-rare diseases, making it
challenging for payers to predict the number of patients who will
be covered within their plans. This innovative approach offers
greater certainty to payers that their overall financial risk will
be adjusted if a substantially larger number of patients than
currently estimated are identified, diagnosed, and treated with
GIVLAARI.
“Patients can sometimes experience lengthy delays waiting for
access when a new medicine becomes available, as payers can be
challenged to determine both which patients may best respond and
the number of potentially undiagnosed patients in their plans,”
said Barry Greene, President of Alnylam Pharmaceuticals. “Our
proactive approach with VBAs is intended to act as ‘insurance for
insurers’ and builds upon our Alnylam Patient Access Philosophy to
do everything we can to accelerate patient access to innovative
medicines at a sustainable rate to the healthcare system. We are
proud of the access achieved in the U.S. for ONPATTRO, and are
committed to seeking innovative approaches for patient access with
GIVLAARI and each of our approved medicines.”
Currently, the population of AHP patients with diagnosed, active
disease in the U.S. and Europe is estimated to be approximately
3,000. Due to the broad, non-specific range of symptoms and
comorbidities experienced by AHP patients, the time to diagnosis
can be as long as 15 years.
"Harvard Pilgrim applauds Alnylam’s efforts to help us manage
plan members’ costs if the number of patients treated exceeds
initial forecasts based upon existing prevalence models,” said
Michael Sherman, M.D., Chief Medical Officer at Harvard Pilgrim.
“This prevalence-based adjustment framework may become a model
approach for ultra-rare diseases where few or no therapies have
previously existed and where diagnosis rates are uncertain.”
“Express Scripts, Accredo, and Cigna are committed to deliver
simpler, more affordable, more predictable ways for patients with
rare diseases to receive appropriate care and treatment,” said
Steve Miller, M.D., Executive Vice President & Chief Clinical
Officer at Express Scripts. “The type of agreement Alnylam is
proposing helps ensure people living with acute hepatic porphyria
have access to breakthrough medicines and that plans will get value
for every dollar they pay for these therapies.”
In the absence of GIVLAARI, an AHP patient can cost $400,000 -
$650,000 annually for treatment of attacks, including
hospitalization, hemin administration, and other medical
interventions. GIVLAARI has been shown to reduce the frequency of
attacks thereby providing the potential to reduce or avoid the
costs of hospitalization and other interventions. Under Alnylam’s
new and enhanced VBA framework, payments will be tied to patient
clinical outcomes in the real-world setting mirroring those
achieved in the ENVISION study in terms of reduced AHP attacks.
Alnylam’s Patient Access Philosophy The new VBA framework
announced today for ultra-rare diseases such as AHP builds upon
Alnylam’s Patient Access Philosophy, first released nearly a year
ahead of the approval of its first RNAi therapeutic. As part of
Alnylam’s Access Philosophy, the Company commits to not increase
the price of GIVLAARI by more than the consumer price index for
urban consumers (CPI-U), a measure of inflation, in the absence of
significant investment associated with a meaningful label
expansion. Commercially insured patients are expected to have
little-to-no out-of-pocket costs for GIVLAARI. To see Alnylam’s
progress as of September 2019, download the report.
Alnylam Assist® Alnylam is deeply committed to helping
patients with AHP get access to GIVLAARI. A comprehensive patient
support services program, Alnylam Assist®, will offer an in-house
team of Case Managers to assist patients with verification of
insurance benefits and financial assistance for those who qualify.
Patients will also be eligible to receive support from Patient
Education Liaisons, who can answer questions about disease and
treatment. Physicians and patients can learn more about Alnylam’s
comprehensive patient services by visiting AlnylamAssist.com or
call 1-833-256-2478.
Visit GIVLAARI.com for more information, including full
Prescribing Information.
GIVLAARI Important Safety Information
Contraindications GIVLAARI is contraindicated in patients
with known severe hypersensitivity to givosiran. Reactions have
included anaphylaxis.
Anaphylactic Reaction Anaphylaxis has occurred with
GIVLAARI treatment (<1% of patients in clinical trials). Ensure
that medical support is available to appropriately manage
anaphylactic reactions when administering GIVLAARI. Monitor for
signs and symptoms of anaphylaxis. If anaphylaxis occurs,
immediately discontinue administration of GIVLAARI and institute
appropriate medical treatment.
Hepatic Toxicity Transaminase elevations (ALT) of at
least 3 times the upper limit of normal (ULN) were observed in 15%
of patients receiving GIVLAARI in the placebo-controlled trial.
Transaminase elevations primarily occurred between 3 to 5 months
following initiation of treatment.
Measure liver function tests prior to initiating treatment with
GIVLAARI, repeat every month during the first 6 months of
treatment, and as clinically indicated thereafter. Interrupt or
discontinue treatment with GIVLAARI for severe or clinically
significant transaminase elevations. In patients who have dose
interruption and subsequent improvement, reduce the dose to 1.25
mg/kg once monthly. The dose may be increased to the recommended
dose of 2.5 mg/kg once monthly if there is no recurrence of severe
or clinically significant transaminase elevations at the 1.25 mg/kg
dose.
Renal Toxicity Increases in serum creatinine levels and
decreases in estimated glomerular filtration rate (eGFR) have been
reported during treatment with GIVLAARI. In the placebo-controlled
study, 15% of patients receiving GIVLAARI experienced a
renally-related adverse reaction. The median increase in creatinine
at Month 3 was 0.07 mg/dL. Monitor renal function during treatment
with GIVLAARI as clinically indicated.
Injection Site Reactions Injection site reactions were
reported in 25% of patients receiving GIVLAARI in the
placebo-controlled trial. Symptoms included erythema, pain,
pruritus, rash, discoloration, or swelling around the injection
site. One (2%) patient experienced a single, transient, recall
reaction of erythema at a prior injection site with a subsequent
dose administration.
Drug Interactions Concomitant use of GIVLAARI increases
the concentration of CYP1A2 or CYP2D6 substrates, which may
increase adverse reactions of these substrates. Avoid concomitant
use of GIVLAARI with CYP1A2 or CYP2D6 substrates for which minimal
concentration changes may lead to serious or life-threatening
toxicities. If concomitant use is unavoidable, decrease the CYP1A2
or CYP2D6 substrate dosage in accordance with approved product
labeling.
Adverse Reactions The most common adverse reactions that
occurred in patients receiving GIVLAARI were nausea (27%) and
injection site reactions (25%).
For additional information about GIVLAARI, please see full
Prescribing Information.
About GIVLAARI™ (givosiran) GIVLAARI is an RNAi
therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for
the treatment of adults with acute hepatic porphyria (AHP). In the
pivotal study, GIVLAARI was shown to significantly reduce the rate
of porphyria attacks that required hospitalizations, urgent
healthcare visits or IV hemin administration at home compared to
placebo. GIVLAARI is Alnylam’s first commercially-available
therapeutic based on its Enhanced Stabilization Chemistry
ESC-GalNAc conjugate technology to increase potency and durability.
GIVLAARI is administered via subcutaneous injection once monthly at
a dose based on actual body weight and should be administered by a
healthcare professional. GIVLAARI works by specifically reducing
elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger
RNA (mRNA), leading to reduction of toxins associated with attacks
and other disease manifestations of AHP. For more information about
GIVLAARI, visit GIVLAARI.com.
About AHP Acute hepatic porphyria (AHP) refers to a
family of ultra-rare, genetic diseases characterized by potentially
life-threatening attacks and, for some patients, chronic
manifestations that negatively impact daily functioning and quality
of life. AHP is comprised of four types: acute intermittent
porphyria (AIP), hereditary coproporphyria (HCP), variegate
porphyria (VP), and ALA dehydratase-deficiency porphyria (ADP).
Each type of AHP results from a genetic defect leading to
deficiency in one of the enzymes of the heme biosynthesis pathway
in the liver. AHP disproportionately impacts women of working and
childbearing age, and symptoms of the disease vary widely. Severe,
unexplained abdominal pain is the most common symptom, which can be
accompanied by limb, back, or chest pain, nausea, vomiting,
confusion, anxiety, seizures, weak limbs, constipation, diarrhea,
or dark or reddish urine. The nonspecific nature of AHP signs and
symptoms can often lead to misdiagnoses of other more common
conditions such as viral gastroenteritis, irritable bowel syndrome
(IBS), addiction withdrawal and appendicitis. Consequently,
patients with AHP can wait up to 15 years for a confirmed
diagnosis. In addition, long-term complications and comorbidities
of AHP can include hypertension, chronic kidney disease or liver
disease, including fibrosis, cirrhosis and hepatocellular
carcinoma.
About RNAi RNAi (RNA interference) is a natural cellular
process of gene silencing that represents one of the most promising
and rapidly advancing frontiers in biology and drug development
today. Its discovery has been heralded as “a major scientific
breakthrough that happens once every decade or so,” and was
recognized with the award of the 2006 Nobel Prize for Physiology or
Medicine. By harnessing the natural biological process of RNAi
occurring in our cells, a new class of medicines, known as RNAi
therapeutics, is now a reality. Small interfering RNA (siRNA), the
molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic
platform, function upstream of today’s medicines by potently
silencing messenger RNA (mRNA) – the genetic precursors – that
encode for disease-causing proteins, thus preventing them from
being made. This is a revolutionary approach with the potential to
transform the care of patients with genetic and other diseases.
About Alnylam Alnylam (Nasdaq: ALNY) is leading the
translation of RNA interference (RNAi) into a whole new class of
innovative medicines with the potential to transform the lives of
people afflicted with rare genetic, cardio-metabolic, hepatic
infectious, and central nervous system (CNS)/ocular diseases. Based
on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach for the treatment of a wide
range of severe and debilitating diseases. Founded in 2002, Alnylam
is delivering on a bold vision to turn scientific possibility into
reality, with a robust RNAi therapeutics platform. Alnylam’s
commercial RNAi therapeutic products are ONPATTRO® (patisiran),
approved in the U.S., EU, Canada, Japan, and Switzerland, and
GIVLAARI™ (givosiran), approved in the U.S. Alnylam has a deep
pipeline of investigational medicines, including five product
candidates that are in late-stage development. Alnylam is executing
on its "Alnylam 2020" strategy of building a multi-product,
commercial-stage biopharmaceutical company with a sustainable
pipeline of RNAi-based medicines to address the needs of patients
who have limited or inadequate treatment options. Alnylam employs
over 1,200 people worldwide and is headquartered in Cambridge, MA.
For more information about our people, science and pipeline, please
visit www.alnylam.com and engage with us on Twitter at @Alnylam or
on LinkedIn.
Alnylam Forward Looking Statements Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam's views with
respect to the approval of GIVLAARI™ (givosiran) injection for
subcutaneous use, and the implications of such approval for
patients and their caregivers, its plans to offer a new and
enhanced framework for VBAs designed to help patients with AHP gain
access to GIVLAARI and , the status of discussions with leading
payers about VBAs for GIVLAARI that would incorporate this
framework, expectations regarding the estimated population of AHP
patients with diagnosed, active disease in the U.S. and Europe, the
potential time to diagnosis for patients and the potential cost to
insurers of treatment in the absence of GIVLAARI, expectations
regarding the potential for GIVLAARI to reduce the frequency of
attacks thereby reducing or avoiding the costs of treatment, its
plan to offer comprehensive support services for people prescribed
GIVLAARI through Alnylam Assist®, and expectations regarding its
“Alnylam 2020” guidance for the advancement and commercialization
of RNAi therapeutics, constitute forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995. Actual results and future plans may
differ materially from those indicated by these forward-looking
statements as a result of various important risks, uncertainties
and other factors, including, without limitation, Alnylam's ability
to discover and develop novel drug candidates and delivery
approaches, successfully demonstrate the efficacy and safety of its
product candidates, the pre-clinical and clinical results for its
product candidates, which may not be replicated or continue to
occur in other subjects or in additional studies or otherwise
support further development of product candidates for a specified
indication or at all, actions or advice of regulatory agencies,
which may affect the design, initiation, timing, continuation
and/or progress of clinical trials or result in the need for
additional pre-clinical and/or clinical testing, delays,
interruptions or failures in the manufacture and supply of its
product candidates, obtaining, maintaining and protecting
intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, including GIVLAARI, progress in establishing a commercial
and ex-United States infrastructure, successfully launching,
marketing and selling its approved products globally, including
GIVLAARI, Alnylam’s ability to successfully expand the indication
for ONPATTRO in the future, competition from others using
technology similar to Alnylam's and others developing products for
similar uses, Alnylam's ability to manage its growth and operating
expenses and achieve a self-sustainable financial profile in the
future, obtain additional funding to support its business
activities, and establish and maintain strategic business alliances
and new business initiatives, Alnylam's dependence on third
parties, including Regeneron, for development, manufacture and
distribution of products, and Ironwood, for assistance with the
education about and promotion of GIVLAARI, the outcome of
litigation, the risk of government investigations, and unexpected
expenditures, as well as those risks more fully discussed in the
"Risk Factors" filed with Alnylam's most recent Quarterly Report on
Form 10-Q filed with the Securities and Exchange Commission (SEC)
and in other filings that Alnylam makes with the SEC. In addition,
any forward-looking statements represent Alnylam's views only as of
today and should not be relied upon as representing its views as of
any subsequent date. Alnylam explicitly disclaims any obligation,
except to the extent required by law, to update any forward-looking
statements.
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version on businesswire.com: https://www.businesswire.com/news/home/20191120005870/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media)
617-682-4340
Joshua Brodsky
(Investors) 617-551-8276
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